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Top Ten Awards

March 5, 2019 - 5:00pm to 8:00pm

National Press Club529 14th Street NWWashington, DC 20045

The Clinical Research Forum is proud to announce the 2019 Top Ten Clinical Research Achievement Awards. All ten outstanding research studies were recognized and honored on March 5, 2019. In addition, three of the studies received further honors:

A Cluster-Randomized Trial of Blood-Pressure Reduction in Black Barbershops, led by Dr. Ron Victor and nominated by UCLA and Cedar-Sinai, receivedThe Herbert Pardes Clinical Research Excellence Award. Named in honor of CR Forum board member Herbert Pardes for his profound impact on clinical research and academic medicine, this award, which comes with a $7,500 cash prize, is for the research study that best shows a high degree of innovation and creativity, advances science, and has an impact upon human disease.

Defuse 3 Study, nominated by Stanford University and represented by Dr. Gregory Albers, andTransthyretin Cardiac Amyloidosis: Overlooked, Under Appreciated and Treatable,nominated by Columbia University and represented by Dr. Mathew S. Maurer both received Distinguished Clinical Research Achievement Awards, presented to the top two studies that show creativity, innovation, or a novel approach that demonstrates an immediate impact on the health and well-being of patients. Each study received a $5,000 cash prize.

The 2019 Top Ten Clinical Research Achievement Awardees are:

A Cluster-Randomized Trial of Blood-Pressure Reduction in Black Barbershops, nominated by the University of California, Los Angeles, and represented by Dr. Eduardo Marbán, Director of the Smidt Heart Institute at Cedars-Sinai Medical Center. This study aimed to develop a blood-pressure control program for non-hispanic black men in the trusted and comfortable environment of their barbershops, where specially-trained pharmacists delivered medication management to black male patrons affected by hypertension. In just six short months, the barbershop study improved the outcomes and control of high blood pressure by over 60 percent of particpants. The collaboration of physicians and shop owners uniquely promoted the integration of hard-to-reach underserved populations into research by increasing awareness and gaining the endorsement of the trusted barbershop owners, who are community members themselves.

Balanced Crystalloids versus Saline in Critically Ill Adults, nominated by Vanderbilt University, and represented by Dr. Matthew W. Semler, Assistant Professor of Medicine. The most common fluids given to patients in the hospital are saline (fluid made up of water and table salt) and balanced crystalloids (fluid made to be similar to the fluid inside the human body). For decades, some doctors have given patients saline and others have given balanced crystalloids, but no one has known which fluid was better for patients. This study found that giving patients balanced crystalloids rather than saline allowed more patients to be able to leave the hospital alive and without kidney problems. The findings have improved medical care in hospitals across the United States.

Bringing innovative physiologic therapy to patients with hereditary rickets, nominated by Yale University and represented by Dr. Thomas O. Carpenter, Professor of Pediatrics and Director, Yale Center for X-linked Hypophosphatemia. X-linked hypophosphatemia (XLH) is the most common cause of heritable rickets. There has not been an approved therapy for the disorder since its identification over 50 years ago. Children in this study were the first to receive a new therapy that targeted the excess production of Fibroblast growth factor 23 (FGF23), a protein that regulates phosphate concentration in the human body. The study served as the pivotal trial leading to the very recent approval of burosumab for the treatment of XLH in children in both the US and Europe. The study is likely to revolutionize the care of children with XLH, providing a simpler and more effective therapy, with the potential long-term advantages of fewer orthopedic surgeries, and fewer complications of phosphate loading.
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Defuse 3 Study, nominated by Stanford University and represented by Dr. Gregory Albers, the Coyote Foundation Professor and Professor of Neurosurgery. This study challenged the concept that brain tissue dies rapidly following the onset of stroke symptoms and that stroke treatment beyond 6 hours after onset was futile. Using an imaging paradigm and novel software program, researchers were able to identify those stroke patients who continue to have salvageable brain tissue many hours after stroke onset. This ability to identify patients who are most likely to benefit from stroke therapies will greatly accelerate the development and testing of new therapeutic options. Hospitals are now changing their stroke protocols to offer a life-saving stroke therapy to many patients who arrive after the standard six-hour treatment window has elapsed.

Hemophilia B Gene Therapy, nominated by University of Pennsylvania and represented by Dr. Lindsey A. George, Assistant Professor of Pediatrics. Hemophilia B is an inherited bleeding disorder characterized by recurrent and spontaneous bleeding that is treated with approximate weekly intravenous administration of the missing factor IX (FIX) protein; this treatment is difficult to adhere to, incompletely effective, and costly. This is the first gene therapy trial to report a clinical cure for hemophilia B patients.

Medical Management of Early Pregnancy Loss, nominated by University of Pennsylvania and represented by Dr. Courtney A. Schreiber, Associate Professor of Obstetrics and Gynecology. One out of four pregnancies ends in early pregnancy loss, or miscarriage. For the pregnant woman, waiting for natural resolution is often slow and never predictable, and surgical management to complete the miscarriage is invasive, time consuming, and costly. This study is the only rigorous, randomized clinical trial for miscarriage management that compares a combination therapy of mifepristone and misoprostol to misoprostol alone. The rate of expedited success was 84 percent among women who received both drugs, and 67 percent for among those who received misoprostol alone. Based on these results, over 700,000 women per year in the U.S. could be spared surgery or prolonged medical treatment by this combination therapy.

Predicting response of patients with chronic lymphocytic leukemia to chimeric antigen receptor (CAR) T cell therapy, nominated by the University of Pennsylvania, represented by Dr. Joseph A. Fraietta, Assistant Professor of Microbiology and Director, Tumor Immunotherapy Laboratory, Center for Cellular Immunotherapies. A common reason for failure of the immune system to eliminate leukemia is that it does not recognize and kill cancer cells. It is now possible to use gene engineering to introduce synthetic molecules, such as chimeric antigen receptors (CARs) into T cells allowing them to recognize and destroy a patient’s own cancer. This study focused on understanding why CAR T cell therapy is highly effective in some patients, but not others. This would allow physicians to select only patients most likely to benefit from treatment and thus improve their quality of life. The long-term goal is to advance the cure rates of adult leukemia without the side effects typically seen with conventional treatments, and ultimately to extend the benefits of CAR T cells to other cancers.

Robust relationship between air quality and infant mortality in Africa, nominated by Stanford University, represented by Marshall Burke, PhD, Assistant Professor of Earth System Science. This study looked at the effects of air pollution on infant mortality in Sub-Saharan Africa and found that, in 2015, exposure to particulate matter led to 400,000 otherwise preventable infant deaths. Researchers compiled data on nearly 1 million births from household surveys across 30 sub-Saharan African countries spanning 2001-2015. By using new satellite-based measures of air pollution, researchers were able to compare the particulate matter each infant was exposed to while in utero and after birth. The results suggest that high particulate matter concentrations were responsible for 22 percent of infant deaths from 2001 to 2015. Moreover, the study found that relatively small decreases in particulate matter concentrations could result in major reductions in mortality. For example, if countries in Sub-Saharan Africa could achieve reductions in pollution exposure similar to wealthy countries, the benefits to infant health could be larger than nearly all current health interventions, such as vaccinations or supplements.

The Undiagnosed Diseases Network, nominated by the National Human Genome Research Institute, represented by Dr. Vandana Shashi, Professor of Pediatrics at Duke University and Kim Leblanc, Associate Director of Research Operations for the Undiagnosed Diseases Coordinating Center based at Harvard University. Rare, novel, and undiagnosed disorders challenge patients, their families, and their clinicians. The goals of the Undiagnosed Diseases Network (UDN) are to develop a model to speed the diagnosis of rare or previously unrecognized diseases, improve care, and stimulate research. The UDN plans to reach these objectives by growing the numbers of experts who can make accurate diagnoses, by determining best practices for introducing the strategy into medical centers nationwide, and by sharing results, data, specimens, and approaches with the scientific and medical communities.

Transthyretin Cardiac Amyloidosis: Overlooked, Under Appreciated and Treatable, nominated by Columbia University and represented by Dr. Mathew S. Maurer, Arnold and Arlene Goldstein Professor of Cardiology. Transthyretin amyloid cardiomyopathy is an under-diagnosed and often overlooked cause of heart failure. This randomized, double blind, prospective, and international multicenter study evaluated the drug tafamidis for patients with the genetic and non-genetic forms of transthyretin amyloidosis. Use of the drug showed a significant reduction in mortality and frequency of cardiovascular-related hospitalizations. Collectively, these findings indicate that tafamidis is the first effective therapy for patients with transthyretin amyloid cardiomyopathy.2